A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: VX-445/TEZ/IVA; Drug: IVA; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03525444

VX17-445-102

2018-000183-28 (EudraCT Number)

Details and patient eligibility

About

This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

Enrollment: 405 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Heterozygous for the F508del mutation (F/MF)
• Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

• Clinically significant cirrhosis with or without portal hypertension
• Lung infection with organisms associated with a more rapid decline in pulmonary status
• Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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405 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Participants who received placebo matched to VX-445/TEZ/IVA for 24 weeks in the TC treatment period.

Treatment:

Drug: Placebo

VX-445/TEZ/IVA TC

Experimental group

Description:

Participants who received VX-445 200 mg/TEZ 100 mg/IVA150 mg as fixed-dose combination (FDC) tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period.

Treatment:

Drug: IVA

Drug: VX-445/TEZ/IVA